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(11)

EP 3 612 624 A1

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(43) Date of publication: 26.02.2020 Bulletin 2020/09 (21) Application number: 18724470.2 (22) Date of filing: 20.04.2018

(51) International Patent Classification (IPC):  C12N 5/0789(2010.01) C12N 5/10(2006.01) A61K 35/15(2015.01) A61K 39/395(2006.01) A61P 35/00(2006.01) C12N 5/0786(2010.01) A61K 31/405(2006.01) A61K 38/21(2006.01) A61K 48/00(2006.01) (86) International application number: PCT/EP2018/060238 (87) International publication number: WO 2018/193119 (25.10.2018 Gazette 2018/43)

(84) Designated Contracting States: AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR Designated Extension States: BA ME Designated Validation States: KH MA MD TN (30) Priority: 21.04.2017 GB 201706410 30.01.2018 GB 201801511 (71) Applicants: Ospedale San Raffaele S.r.l. 20132 Milano (IT) Fondazione Telethon 00185 Roma (IT)	(72) Inventors: NALDINI, Luigi 20132 Milan (IT) ESCOBAR, Giulia 20132 Milan (IT) GENTNER, Bernhard Rudolf 20132 Milan (IT) MUCCI, Adele 20132 Milan (IT) (74) Representative: D Young & Co LLP  120 Holborn London EC1N 2DY London EC1N 2DY (GB)